Not so fast, Mr Pres­i­dent: Trump's plan for low­er­ing pre­scrip­tion drug prices hits a road­block, as Cana­da bans bulk drug ex­ports

A new rule that took ef­fect Mon­day paves the way for Pres­i­dent Don­ald Trump to ful­fill his promise of let­ting states im­port cer­tain pre­scrip­tion drugs from Cana­da — but our neigh­bor up north is say­ing ‘not so fast.’

On Sat­ur­day, the Cana­di­an gov­ern­ment is­sued new mea­sures to pre­vent bulk ex­ports to the US that could wors­en drug short­ages across the coun­try. Cana­da sources 68% of its drugs in­ter­na­tion­al­ly, ac­cord­ing to a state­ment from Min­is­ter of Health Pat­ty Ha­j­du. The coun­try has re­peat­ed­ly tak­en the stance that the US’ im­por­ta­tion rule wouldn’t be ef­fec­tive in low­er­ing pre­scrip­tion drug prices, as its own mar­ket is so small.

“The need for vig­i­lance in main­tain­ing the na­tion­al drug sup­ply con­tin­ues,” Ha­j­du’s state­ment read.

Start­ing Sat­ur­day, cer­tain drugs in­tend­ed for the Cana­di­an mar­ket are banned from dis­tri­b­u­tion out­side the coun­try if the sale “would cause or wors­en a drug short­age.” Com­pa­nies al­so have to pro­vide in­for­ma­tion to “as­sess ex­ist­ing or po­ten­tial short­ages” when re­quest­ed, and with­in 24 hours if there’s a se­ri­ous risk.

The rule is part of Trump’s last-ditch ef­fort to low­er pre­scrip­tion drug prices for Amer­i­cans, some­thing he’s re­peat­ed­ly promised to do. The pres­i­dent signed an ex­ec­u­tive or­der back in Ju­ly call­ing for the FDA to com­plete the “rule­mak­ing process” to al­low the im­por­ta­tion of Cana­di­an pre­scrip­tion drugs. And last week, he an­nounced plans to im­ple­ment some­thing called the “most fa­vored na­tions” rule, which would tie prices in the US to those in oth­er de­vel­oped coun­tries and end cer­tain re­bates paid to mid­dle­men.

That rule, op­posed by phar­ma com­pa­nies, would come in­to ef­fect in Jan­u­ary, Trump said. It would ap­ply to 50 cost­ly Medicare Part B med­i­cines. The plan is to phase in the low­est price in oth­er coun­tries and “blend” it with the av­er­age sales price, then add a flat amount per dose for each ap­plic­a­ble drug.

In the mean­time, Trump said he’d al­low states to pur­chase cheap­er drugs from Cana­da. But it could be a while be­fore cheap­er drugs make their way across the bor­der — if ever. In its fi­nal rule is­sued back in Sep­tem­ber, the FDA said states, ter­ri­to­ries, tribes, phar­ma­cists and whole­salers would need to set up new pro­grams and sub­mit ap­pli­ca­tions to im­port drugs, which would have to meet sev­er­al spec­i­fi­ca­tions. That would in­clude re­la­bel­ing and test­ing the drugs for “au­then­tic­i­ty and degra­da­tion,” ac­cord­ing to the FDA. And the pro­grams would have to demon­strate “sig­nif­i­cant cost re­duc­tions” to the con­sumer.

“If you put Cana­di­an drugs on a dogsled and point­ed it in the di­rec­tion of Flori­da, the dogs would ar­rive long be­fore any drugs through this reg­u­la­to­ry pro­pos­al,” for­mer HHS of­fi­cial Chris Meekins told the Wash­ing­ton Post back in De­cem­ber.

Un­der a law es­tab­lished in 2003, such im­por­ta­tion is on­ly al­lowed if cer­ti­fied by the HHS sec­re­tary — and Alex Azar is the first to for­mal­ly do so.

“Our health care sys­tem is a sym­bol of our na­tion­al iden­ti­ty and we are com­mit­ted to de­fend­ing it. The ac­tions we are tak­ing to­day will help pro­tect Cana­di­ans’ ac­cess to the med­ica­tion they re­ly on,” Ha­j­du said in a state­ment.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Ciaran McCrickard)

An un­ortho­dox pro­pos­al for Bio­gen's Medicare-man­dat­ed Aduhelm tri­al

Biogen has gone full blitz since Medicare announced it would only cover its new Alzheimer’s drug when used in clinical trials, accusing the agency of discriminating against Alzheimer’s patients and trying to get physicians to change regulators’ minds.  Critics, meanwhile, cheered what they see as a necessary wall protecting payers and patients from an unproven and unsafe drug.

Far less attention, though, has gone to what a Medicare-funded clinical trial would actually look like. Biogen has operated as if it would be a standard late-stage Alzheimer’s trial, enrolling a couple thousand patients and giving half placebo.

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